KR20210108423A - 아데노 관련 바이러스 (aav) 생산자 세포주 및 관련 방법 - Google Patents

아데노 관련 바이러스 (aav) 생산자 세포주 및 관련 방법 Download PDF

Info

Publication number
KR20210108423A
KR20210108423A KR1020217022728A KR20217022728A KR20210108423A KR 20210108423 A KR20210108423 A KR 20210108423A KR 1020217022728 A KR1020217022728 A KR 1020217022728A KR 20217022728 A KR20217022728 A KR 20217022728A KR 20210108423 A KR20210108423 A KR 20210108423A
Authority
KR
South Korea
Prior art keywords
promoter
gene
mammalian cell
nucleic acid
control
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
KR1020217022728A
Other languages
English (en)
Korean (ko)
Inventor
빙난 구
케이틀린 엠. 겐터
아난디타 세스
Original Assignee
론자 워커스빌 아이엔씨.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 론자 워커스빌 아이엔씨. filed Critical 론자 워커스빌 아이엔씨.
Publication of KR20210108423A publication Critical patent/KR20210108423A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
    • C12N2830/006—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB tet repressible
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020217022728A 2018-12-21 2019-12-18 아데노 관련 바이러스 (aav) 생산자 세포주 및 관련 방법 Pending KR20210108423A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862783589P 2018-12-21 2018-12-21
US62/783,589 2018-12-21
US201962866092P 2019-06-25 2019-06-25
US62/866,092 2019-06-25
PCT/US2019/067156 WO2020132059A1 (en) 2018-12-21 2019-12-18 Adeno-associated virus (aav) producer cell line and related methods

Publications (1)

Publication Number Publication Date
KR20210108423A true KR20210108423A (ko) 2021-09-02

Family

ID=71099162

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217022728A Pending KR20210108423A (ko) 2018-12-21 2019-12-18 아데노 관련 바이러스 (aav) 생산자 세포주 및 관련 방법

Country Status (8)

Country Link
US (2) US11739347B2 (https=)
EP (1) EP3891290A4 (https=)
JP (2) JP7650800B2 (https=)
KR (1) KR20210108423A (https=)
CN (1) CN113396222A (https=)
IL (1) IL284219A (https=)
SG (1) SG11202106217TA (https=)
WO (1) WO2020132059A1 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4189077A1 (en) * 2020-07-30 2023-06-07 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
EP4229205B1 (en) * 2020-10-15 2025-05-14 F. Hoffmann-La Roche AG Nucleic acid constructs for va rna transcription
CN112501209B (zh) * 2020-12-07 2024-02-13 和元生物技术（上海）股份有限公司 外源基因可控表达的腺相关病毒包装方法
CN116829725A (zh) * 2021-02-12 2023-09-29 富士胶片株式会社 用于产生腺相关病毒的试剂盒及其利用
EP4322975A4 (en) * 2021-04-14 2025-03-26 Capsida, Inc. PLASMIDS AND PROCESSES FOR PRODUCING ADENO-ASSOCIATED VIRUSES
EP4410971A4 (en) * 2021-09-30 2025-06-04 FUJIFILM Corporation METHOD FOR PRODUCING AN ADENO-ASSOCIATED VIRUS, CELLS AND EXPRESSION VECTOR
AU2022373653A1 (en) * 2021-10-18 2024-05-02 Regeneron Pharmaceuticals, Inc. Eukaryotic cells comprising adenovirus-associated virus polynucleotides
EP4473123A4 (en) * 2022-02-01 2026-03-11 Shape Therapeutics Inc STABLE CELL LINES FOR INDUCIBLE VAAR VIRION PRODUCTION
EP4491717A4 (en) 2022-03-08 2025-11-12 Fujifilm Corp PRODUCING CELL, PRODUCTION METHOD FOR PRODUCING PRODUCING CELLS AND PRODUCTION METHOD FOR ADENE-ASSOCIATED VIRUS
CN120092093A (zh) * 2022-08-25 2025-06-03 富士胶片株式会社 靶病毒的制造方法及培养组合物
EP4624584A1 (en) * 2022-12-27 2025-10-01 Synplogen Co., Ltd. Helper gene control
US12410436B2 (en) * 2023-10-31 2025-09-09 Lonza Houston, Inc. Adeno-associated virus (AAV) producer cell lines
WO2025122676A1 (en) * 2023-12-05 2025-06-12 Shape Therapeutics Inc. Systems and methods for raav production using a biphasic culture
WO2025168663A1 (en) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles
WO2025252480A1 (en) 2024-06-07 2025-12-11 F. Hoffmann-La Roche Ag Method for purifying plasmid dna
CN118460545B (zh) * 2024-07-08 2025-02-11 凌意(杭州)生物科技有限公司 一种诱导型表达Rep多肽的表达框
US20260022401A1 (en) 2024-07-22 2026-01-22 Hoffmann-La Roche Inc. Novel AAV rep ORFs and Rep polypeptides
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles
WO2026044087A1 (en) * 2024-08-21 2026-02-26 Shape Therapeutics Inc. Increased cellular stability for aav production

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US6037177A (en) * 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production
JP2002153278A (ja) * 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
AU2002305778A1 (en) * 2001-05-31 2002-12-09 The Rockefeller University Method for generating replication defective viral vectors that are helper free
AU2002367943A1 (en) * 2001-12-18 2003-12-22 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
EP1844148A2 (en) 2005-01-31 2007-10-17 University of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
US20130023033A1 (en) * 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
JP6661544B2 (ja) 2014-04-24 2020-03-11 ミルテニイ バイオテック ゲゼルシャフト ミット ベシュレンクテル ハフツング 遺伝子改変したｔ細胞の自動生成法
JP2018508223A (ja) * 2015-03-16 2018-03-29 ボード・オブ・トラスティーズ、サザン・イリノイ・ユニバーシティ 安定な遺伝子発現のための系
WO2018136566A1 (en) 2017-01-18 2018-07-26 F1 Oncology, Inc. Methods of transducing and expanding immune cells and uses thereof
US10858631B2 (en) * 2017-04-18 2020-12-08 Glaxosmithkline Intellectual Property Development Limited Methods for adeno-associated viral vector production

Also Published As

Publication number Publication date
JP7650800B2 (ja) 2025-03-25
CN113396222A (zh) 2021-09-14
US11739347B2 (en) 2023-08-29
EP3891290A1 (en) 2021-10-13
JP2022516004A (ja) 2022-02-24
US20240018545A1 (en) 2024-01-18
IL284219A (en) 2021-08-31
WO2020132059A1 (en) 2020-06-25
EP3891290A4 (en) 2022-11-02
US12529074B2 (en) 2026-01-20
SG11202106217TA (en) 2021-07-29
US20200199627A1 (en) 2020-06-25
JP2025102801A (ja) 2025-07-08

Similar Documents

Publication Publication Date Title
KR20210108423A (ko) 아데노 관련 바이러스 (aav) 생산자 세포주 및 관련 방법
US20230235010A1 (en) Compositions and methods for tcr reprogramming using fusion proteins
AU2019203955C1 (en) Multipartite signaling proteins and uses thereof
KR102319845B1 (ko) 조류 숙주 세포에 대한 crispr-cas 시스템
KR102755022B1 (ko) 아데노바이러스 벡터 및 이의 용도
KR101666228B1 (ko) 생물치료학적 분자를 발현시키기 위한 치료학적 유전자-스위치 작제물 및 생물반응기, 및 이의 용도
CN101605899B (zh) 用于大量生产重组蛋白质的新载体和表达细胞系以及使用其生产重组蛋白质的方法
US20030119104A1 (en) Chromosome-based platforms
KR20230019063A (ko) C9orf72 연관 질환의 치료를 위한 삼중 기능 아데노-연관 바이러스 (aav) 벡터
KR20210105382A (ko) 단백질을 코딩하는 rna
KR20240021906A (ko) 발현 벡터, 박테리아 서열-무함유 벡터, 및 이를 제조하고 사용하는 방법
CN114026242A (zh) 具有髓鞘蛋白零启动子的aav载体及其用于治疗雪旺细胞相关疾病如charcot-marie-tooth疾病的用途
KR20230117327A (ko) 가용성 알칼리성 포스파타제 작제물 및 가용성 알칼리성 포스파타제 작제물을 인코딩하는 폴리뉴클레오티드를 포함하는 발현 벡터
CN112877292A (zh) 产生人抗体的细胞
AU2017233862B2 (en) Methods and compositions for increased double stranded RNA production
CN114786685B (zh) 一种基于线粒体的药物递送系统及其用途
KR20240022571A (ko) Rna-가이드된 이펙터 동원을 위한 시스템, 방법 및 성분
CN114807140B (zh) 一种肌源性细胞血糖响应型表达sia的启动子、重组载体及其构建方法和应用
CN117881788A (zh) 表达载体、无细菌序列载体及其制备和使用方法
HK40049685A (en) Adeno-associated virus (aav) producer cell line and related methods
KR102393402B1 (ko) 세포 내 존재 단백질과 세포 외부로 분비되는 단백질을 동시 발현하는 이중발현벡터를 포함하는 암의 예방 또는 치료용 조성물
HK40111561A (zh) 表达载体、无细菌序列载体及其制备和使用方法
CA2522166C (en) Lambda integrase mutein for use in recombination
TW202309287A (zh) 調節基因表現之組合物及方法
HK40091834A (zh) 增加双链rna产生的方法和组合物

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

D13-X000 Search requested

St.27 status event code: A-1-2-D10-D13-srh-X000

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000